Monday, December 10, 2018
   
Text Size

Site Search powered by Ajax

New drug offers hope for treating Huntington's disease

Researchers at UCL fix a protein defect that causes devastating genetic disease Huntington's in patients for first time.

brain

An experimental drug has for the first time been shown to slow the progression of Huntington's disease, giving hopes to patients suffering from the incurable disorder.

Scientists at UK's University College of London said on Monday that an early-stage trial found that the treatment had "successful lowered" the levels of a toxic protein that causes the devastating genetic disease.

Sarah Tabrizi, a professor of clinical neurology who led the UCL research, called the ability of the drug, IONIS-HTT(Rx), to lower the mutant huntingtin protein "ground-breaking".

"The key now is to move quickly to a larger trial to test whether IONIS-HTT(Rx) slows disease progression," she said. 

What is Huntington's disease?

Huntington's is a disorder that causes irreversible damage to the brain, affecting patients' movements, behaviour and ability to think clearly.

It is caused by an inherited defect in a single section of DNA called the huntingtin gene.

Most people with Huntington's develop symptoms in their early 30s or 40s and die around 10 to 20 years after symptoms start.

There is currently no treatment to stop or reverse the disease, and existing medication can only help manage its symptoms.

Landmark trial

The UCL trial involved 46 patients from the UK, Germany and Canada.

They were each given four spinal injections of either the drug or a placebo. 

UCL said the drug produced "significant, dose-dependent lowering of the level of mutant huntingtin" and marks the first time that the protein has been lowered in the nervous systems of patients.

IONIS-HTT(Rx) works by effectively silencing the defective huntingtin gene. 

Professor John Hardy, an expert on diseases affecting the nervous system, told the BBC that the trial was "potentially, the biggest breakthrough in neurodegenerative disease in the past 50 years". 

Ionis Pharmaceuticals, the company behind the drug, said in a statement on Monday that following the trial pharmaceutical giant Roche had exercised its right to license IONIS-HTT(Rx) at a cost of $45m.

The prevalence of the disease varies by ethnicity and geographical location. In Europe, it is estimated that about five to seven people in 100,000 are affected.

About 8,500 people in the UK have Huntington's, while 30,000 people have it in the US.


blog comments powered by Disqus

Subscribe via RSS or Email:

Two border crossings open in divided Cyprus, first in 8 years

Read More

Ukraine rebel regions vote in polls slammed by Kiev and the West

Read More

UN chief sees 'parallels' with 1930s amid nationalism warnings

Read More

Macron urges world leaders to 'fight for peace' on WWI centenary

Read More

Erdogan and Trump meet in Paris amid Khashoggi crisis

Read More

12 killed in Sicily as death toll in Italy floods mounts

Read More

Most Read Articles

Most Read News

Donation

Thanks to all of our supporters for your generosity and your encouragement of an independent press!

Enter Amount:

Featured_Author

Login






Login reminder Forgot login?

Subscribe to MWC News Alert

Email Address

Subscribe in a reader Facebok page Twitter page

Israel pounds Gaza

India's Kerala state devastated

Capturing life under apartheid